Therapeutic Indications

Blood and Bone Marrow Cancers

Blood cancers affect the production and function of blood cells. Most of these cancers start in the bone marrow where blood is produced. Stem cells in the bone marrow mature and develop into three types of blood cells: red blood cells, white blood cells, or platelets. In most blood cancers, the normal blood cell development process is interrupted by uncontrolled growth of an abnormal type of blood cells. These abnormal blood cells, or cancerous cells, prevent blood from performing many of its functions, like fighting off infections or preventing serious bleeding.


The three main types of blood cancers are leukemia, lymphoma and myeloma. Leukemia a type of cancer of bone marrow, is caused by the proliferation of abnormal cells in the bone marrow. Lymphoma is a type of blood cancer that affects the lymphoid organs where are located the immune cells. Myeloma is a cancer of bone marrow due to the proliferation of abnormal plasma cells, which are cells producing the antibodies.

Acute Lymphoblastic Leukemia (ALL)

ALL is a heterogeneous hematologic disease characterized by the proliferation of immature lymphoid cells in the bone marrow, peripheral blood, and other organs. The proliferation and accumulation of blast cells in the marrow results in suppression of hematopoiesis and, thereafter, anemia, thrombocytopenia, and neutropenia. ALL represents 75% to 80% of acute leukemia among children, making it the most common form of childhood leukemia; by contrast, ALL represents approximately 20% of all leukemia among adults. The cure rates and survival outcomes for patients with ALL have improved dramatically over the past several decades, primarily among children. Improvements are largely owed to advances in the understanding of the molecular genetics and pathogenesis of the disease, the incorporation of risk-adapted therapy, and the advent of new targeted agents. Despite great progress in the development of curative therapies, ALL remains a leading cause of pediatric cancer-related mortality for patients presenting with a relapsed or refractory disease. New therapies are needed to overcome chemotherapy resistance and reduce non-specific treatment associated side effects.

T-Acute Lymphoblastic Leukemia (T-ALL)

T-ALL is an aggressive malignant neoplasm of the bone marrow. It affects the lymphoid-cell-producing stem cells, in particular a type of white blood cell called T lymphocytes as opposed to B-acute lymphoblastic leukemia (B-ALL) which affects B lymphocytes. It accounts for approximately 20% of all cases of ALL and is more common in adults than children. The diagnosis and treatment of T-ALL remains a challenge with its uncommon and aggressive presentation.

Acute Myeloid Leukemia (AML)

AML is characterized by infiltration of the bone marrow, blood, and other tissues by proliferative, clonal, abnormally and/or poorly differentiated cells of the hematopoietic system called blast cells. These cells interfere with normal hematopoiesis, thus contributing to the bone marrow failure which is the most common underlying cause of death. Although it can occur in children and adults, AML is primarily a disease of the elderly, with an incidence of 15 cases per 100,000 for those over 60 and a median age of patients with AML of 67 years. While complete response rates can be as high as 80% in patients undergoing initial induction cytotoxic chemotherapy, the majority of AML patients will ultimately be diagnosed with relapsed or refractory disease with a poor prognosis. CD123 is highly expressed on acute myeloid leukemia (AML) leukemic stem cells and blast cells, as well as in other hematologic malignancies, and constitutes an attractive target for AML.

Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

BPDCN is a rare and aggressive hematological neoplasm characterized by the clonal proliferation of precursors of plasmacytoid dendritic cells. The disease may occur at any age, but most patients are elderly men who present with skin lesions and/or involved lymph nodes, spleen, and bone marrow. The few available data reported indicate that its overall incidence is extremely low, accounting for 0.44% of all hematologic malignancies and 0.7% of cutaneous lymphomas. Moreover, the leukemic form of disease is a rare phenomenon, representing fewer than 1% of cases of acute leukemia. Given its rarity and only recent recognition as a distinct clinico-pathological entity, no standardized therapeutic approach has been established for BPDCN and the optimal therapy remains to be defined.

Multiple Myeloma (MM)

MM is a clonal plasma cell malignant neoplasm characterized by the proliferation of a single clone of plasma cells producing a monoclonal immunoglobulin. This clone of plasma cells proliferates in the bone marrow and often results in extensive skeletal destruction with osteolytic lesions, osteopenia, and/or pathologic fractures. MM accounts for approximately 10% of hematologic malignant disorders. The annual incidence, age-adjusted to the US population, is 4.3 per 100,000, resulting in over 20,000 new patients in the United States each year. The median age at onset is 66 years. Nine drugs have been approved over the past fifteen years for the treatment of MM, substantially expanding the number of treatment regimens available for patients in all stages of the disease. In the last decade, survival of multiple myeloma (MM) patients has markedly improved with a median survival of approximately 5 to 7 years but with major variation depending on host factors, stage of the disease, cytogenetic abnormalities, and response to therapy. However, despite this progress, patients with disease refractory to both immunomodulatory drugs (IMiDs) and proteasome inhibitors have a median overall survival (OS) of only 9 months.